AVROBIO to Present Clinical and Preclinical Data at the ASGCT 24th Annual Meeting
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that two oral presentations and two posters featuring data from its pipeline of lysosomal disorder gene therapy programs will be shared at the 24th virtual Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, 2021.
Accepted abstracts will include new data points from an investigator-sponsored Phase 1/2 trial1 in cystinosis and from AVROBIO’s preclinical program in Pompe disease. Encore data from AVROBIO’s clinical programs in Fabry disease and Gaucher disease type 1 will also be presented.
The presentations are listed below and the full preliminary program is available online at the ASGCT website.
LIVE ORAL PRESENTATIONS:
“Long-term hematopoietic stem cell lentiviral gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice”
Tuesday, May 11, 2021, 6:30-6:45 p.m. ET
Niek van Til, Ph.D., senior director, gene therapy, AVROBIO, will present new preclinical data for AVR-RD-03, a gene therapy for Pompe disease, and answer questions in a live Q&A.
“AVR-RD-01, an investigational lentiviral gene therapy for Fabry disease: Clinical data trends from Phase 1 and Phase 2 studies up to 3.5 years”
Friday, May 14, 2021, 12:30 p.m. - 12:45 p.m. ET
Mark Thomas, M.D., principal investigator of the AVROBIO-sponsored FAB-GT Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease, nephrologist at the Department of Nephrology, Royal Perth Hospital and clinical professor at the University of Western Australia Medical School, will present encore data and answer questions in a live Q&A.
RECORDED POSTER PRESENTATIONS:
“Hematopoietic stem cell lentiviral gene therapy for cystinosis: Updated results from a Phase 1/2 clinical trial”
Stephanie Cherqui, Ph.D., principal investigator of the Phase 1/2 clinical trial of AVR-RD-04 (CTNS-RD-04), an investigational gene therapy for cystinosis, and associate professor of pediatrics at University of California, San Diego, School of Medicine, will present new data on the third patient dosed in the trial and recap existing data on the first two patients dosed.